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Avapritinib - Blueprint Medicines

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Drug Profile

Avapritinib - Blueprint Medicines

Alternative Names: AYVAKIT; AYVAKYT; Ayvakyt; BLU-285; CS 3007; Taijihua

Latest Information Update: 10 Mar 2026

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At a glance

  • Originator Blueprint Medicines
  • Developer Blueprint Medicines; CStone Pharmaceuticals; Neopharm Ltd
  • Class 2 ring heterocyclic compounds; Amines; Antineoplastics; Pyrazoles; Pyrimidines; Pyrroles; Skin disorder therapies; Small molecules; Triazines
  • Mechanism of Action Platelet-derived growth factor alpha receptor antagonists; Proto-oncogene protein c-kit inhibitors
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Systemic mastocytosis; Gastrointestinal stromal tumours
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Gastrointestinal stromal tumours; Systemic mastocytosis
  • Phase II Haematological malignancies
  • Phase I/II Solid tumours

Most Recent Events

  • 28 Feb 2026 Updated efficacy and adverse events data from the phase II PIONEER trial in Systemic mastocytosis released by Blueprint Medicines
  • 06 Dec 2025 Updated pharmacodynamics data from the phase II PIONEER trial in Systemic mastocytosis presented at the 67th American Society of Hematology Annual Meeting and Exposition (ASH-HEM-2025)
  • 06 Dec 2025 Efficacy and adverse events data from the phase II PIONEER trial in Systemic mastocytosis presented at the 67th American Society of Hematology Annual Meeting and Exposition (ASH-Hem-2025)

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